My recent poston the push for National Right-To-Try (RTT) including a detailed comment from ISSCR stirred some animated feedback.

In the spirit of encouraging diverse discussion, here I am posting a striking comment I received from a stem cell industry insider regarding that post on RTT as it relates to stem cells and s/he was especially focused on the controversial new Texas stem cell law:

“I understand we disagree about these things, but still a little disappointed in how prominent the “snake oil” salesman scare tactic was. I know it wasn’t you saying these things also, but merely lending your platform. Still; the Texas law is very clear that this can only be accessed after IRB approval AND it has to be a hospital or Medical school IRB. Additionally, the treatment must be administered at that hospital or medical school (state chartered). So it hardly lends itself to the kind of fraud they are talking about. This type of IRB approval and institutional only administration is a greater protection than patients get with FDA APPROVED drugs being prescribed off label. In addition, the criminal penalty for violating the law is clear and enforceable; if you DON”T have the IRB approval and administer it through the hospital or medical school, you can be prosecuted. This is a MUCH more effective tool for going after the frauds than exists without it. I have spoken with DA offices in many states and it’s very difficult to go after these guys. This type of enforcement mechanism is a good step in helping law enforcement; there is no “grey” area.. you either have the med school or you don’t. Texas (not my cup of tea in almost anything else they do) should be praised here for making quantitative steps towards getting the fraud in this area under control; not criticized…”

6 Comments

Thumbs up on the outreach. Putting issues up on The Niche that stir debate and add to the public dissemination of information and opinion is important.

I’d like to see more insiders and stakeholders participate in comments in order to provide the necessary repository of perspectives and valuable feedback. Anonymously if need be. Obviously being a popular research scientist cum blogger allows for a podium of sorts re: subjectivity – that I think is ok and natural. Regularly engaging in discussion with the community and playing the objective moderator will provide for an active forum on the topics, which I would say is in the interest of the public. This I think will greatly assist sector communication.

My personal opinion on the premise of RTT is not unlike most advocates that support science as a means to help allievate suffering. No system is perfect but adapting to and embracing change that will generate more data within a professional context, in as safe and urgent a manner as possible, considering the patient, isn’t fundamentally wrong imo.

The common ground I see here is to develop further the Federal Expanded Access provisions to specifically embrace Industry, instead of alienating them, in support of Doctors and Patients seeking potential solutions, within a professionally sanctioned arena.

As I scientist, I listen carefully to these debates but always come back to the question, what evidence is necessary for safety and efficacy before RTT can be implemented? In the clear case of a classical phase I or phase II study of a drug there are clear studies that must be executed but with RTT I lose any grasp on how “appropriate” the potential treatment is. Is there an independent expert body to review data or can anyone put together a rationale, similar to those you do for animal experiments?

I’ve discussed RTT extensively at both my blogs. Basically, all these laws say is that the experimental intervention must have passed phase I tests and still be in “active development,” whatever that means. I’ve interpreted that to mean that further clinical trials are planned. Of course, only having passed phase I studies is an incredibly low bar to pass. Worse, RTT advocates frequently claim that only drugs shown to be safe are allowed. Of course, no one who knows what he’s talking about would ever say that a drug that’s only passed phase I trials has been shown to be safe, and anyone who does know what he’s talking about who said something so ridiculous would clearly be lying.

1. If IRB approval is required then how does a legitimate IRB review an unapproved therapy which is not being conducted under an IND/IDE?
2. How are costs for these therapies being determined and presumably the patient has to pay as there is no insurance coverage for these unapproved therapies.

I firmly believe that the Scientific community need to find a way to provide access to stem cell therapies but this Texas legislation does not seem to be anywhere near the solution.